What are the indications for recombinant human growth hormone (rhGH) therapy in children?

Updated: Jan 24, 2019
  • Author: Sunil Kumar Sinha, MD; Chief Editor: Robert P Hoffman, MD  more...
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Answer

The commercial introduction of recombinant human growth hormone (rhGH) in 1985 dramatically changed the field of therapy for growth hormone (GH).  Since then, rhGH has been administered to tens of thousands of children worldwide, making it one of the most extensively studied therapies in the pediatric pharmacopoeia. 

The initial GH replacement therapy limited to GH-deficient patients has evolved into a pharmacologic therapy to include different conditions of non-GH deficient short stature. [12]  US Food and Drug Administration (FDA)–approved indications for the administration of rhGH in children include the following:

The European Medicines Agency (EMA) has also approved rhGH for all the above indications except idiopathic short stature (ISS) and Noonan syndrome. 

While available evidence suggests that long-term GH therapy reduces the adult height deficit in children with ISS, questions remain as to whether the impact of the height gained on physical and psychosocial well-being outweigh the burden for patients and parents, potential adverse effects, cost of therapy, and patients’/parents’ expectations. [12]


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